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The ICH E9 (R1) Estimands Guidance1 terminology does not completely address the conceptual needs of time-to-event estimands in the complex oncology context. We previously described how censoring and censoring mechanisms for time-to-event…

Applications · Statistics 2022-03-07 Jonathan M Siegel , Hans-Jochen Weber , Stefan Englert

A Real-World Evidence (RWE) Scientific Working Group (SWG) of the American Statistical Association Biopharmaceutical Section (ASA BIOP) has been reviewing statistical considerations for the generation of RWE to support regulatory…

Applications · Statistics 2023-07-04 Jie Chen , Daniel Scharfstein , Hongwei Wang , Binbing Yu , Yang Song , Weili He , John Scott , Xiwu Lin , Hana Lee

We develop a new framework for evaluating the total policy effect in regression discontinuity designs (RDD), incorporating both the direct effect of treatment on outcomes and the indirect effect arising from distortions in the running…

Econometrics · Economics 2025-12-01 Moyu Liao

In the presence of competing events, many investigators are interested in a direct treatment effect on the event of interest that does not capture treatment effects on competing events. Classical survival analysis methods that treat…

Methodology · Statistics 2025-10-29 Takuya Kawahara , Sean McGrath , Jessica G Young

The estimand framework included in the addendum to the ICH E9 guideline facilitates discussions to ensure alignment between the key question of interest, the analysis, and interpretation. Therapeutic knowledge and drug mechanism play a…

Other Quantitative Biology · Quantitative Biology 2023-04-17 Steven Sun , Hans-Jochen Weber , Emily Butler , Kaspar Rufibach , Satrajit Roychoudhury

Case-cohort studies are conducted within cohort studies, wherein collection of exposure data is limited to a subset of the cohort, leading to a large proportion of missing data by design. Standard analysis uses inverse probability weighting…

Imputation of missing attribute values in medical datasets for extracting hidden knowledge from medical datasets is an interesting research topic of interest which is very challenging. One cannot eliminate missing values in medical records.…

Databases · Computer Science 2016-03-11 Yelipe UshaRani , P. Sammulal

Missing data is unavoidable in longitudinal clinical trials, and outcomes are not always normally distributed. In the presence of outliers or heavy-tailed distributions, the conventional multiple imputation with the mixed model with…

Methodology · Statistics 2022-03-22 Siyi Liu , Yilong Zhang , Gregory T Golm , Guanghan , Liu , Shu Yang

Estimands can help to clarify the research questions being addressed in randomised trials. Because the choice of estimand can affect how relevant trial results are to patients and other stakeholders, such as clinicians or policymakers, it…

Missing data is a systemic problem in practical scenarios that causes noise and bias when estimating treatment effects. This makes treatment effect estimation from data with missingness a particularly tricky endeavour. A key reason for this…

Machine Learning · Statistics 2023-02-27 Jeroen Berrevoets , Fergus Imrie , Trent Kyono , James Jordon , Mihaela van der Schaar

Randomized controlled trials (RCTs) are widely regarded as the gold standard for causal inference in biomedical research. For instance, when estimating the average treatment effect on the treated (ATT), a doubly robust estimation procedure…

Methodology · Statistics 2025-09-26 Chi-Shian Dai , Chao Ying , Yang Ning , Jiwei Zhao

In this article, we present a methodology which takes as input a collection of retracted articles, gathers the entities citing them, characterizes such entities according to multiple dimensions (disciplines, year of publication, sentiment,…

Digital Libraries · Computer Science 2022-10-12 Ivan Heibi , Silvio Peroni

Confounding is a significant obstacle to unbiased estimation of causal effects from observational data. For settings with high-dimensional covariates -- such as text data, genomics, or the behavioral social sciences -- researchers have…

Artificial Intelligence · Computer Science 2024-02-01 Katherine A. Keith , Sergey Feldman , David Jurgens , Jonathan Bragg , Rohit Bhattacharya

Inference about treatment effects for time-to-event outcomes is often obscured by the presence of competing events. A particularly complex situation arises when the treatment influences the occurrence of the competing event. A comprehensive…

The problem of missingness in observational data is ubiquitous. When the confounders are missing at random, multiple imputation is commonly used; however, the method requires congeniality conditions for valid inferences, which may not be…

Methodology · Statistics 2020-07-10 Nathan Corder , Shu Yang

Dropout poses a significant challenge to causal inference in longitudinal studies with time-varying treatments. However, existing research does not simultaneously address dropout and time-varying treatments. We examine selective…

Methodology · Statistics 2025-03-18 Zhichao Jiang , Eli Ben-Michael , D. James Greiner , Ryan Halen , Kosuke Imai

Patients often discontinue treatment in a clinical trial because their health condition is not improving. Consequently, the patients still in the study at the end of the trial have better health outcomes on average than the initial patient…

Methodology · Statistics 2022-06-06 Alex Ocampo , Heinz Schmidli , Peter Quarg , Francesca Callegari , Marcello Pagano

Motivated by the study of state opioid policies, we propose a novel approach that uses autoregressive models for causal effect estimation in settings with panel data and staggered treatment adoption. Specifically, we seek to estimate the…

When fitting a generalized linear model -- such as a linear regression, a logistic regression, or a hierarchical linear model -- analysts often wonder how to handle missing values of the dependent variable Y. If missing values have been…

Methodology · Statistics 2017-03-27 Paul T. von Hippel

Missing attribute values are quite common in the datasets available in the literature. Missing values are also possible because all attributes values may not be recorded and hence unavailable due to several practical reasons. For all these…

Information Retrieval · Computer Science 2016-05-04 Yelipe UshaRani , P. Sammulal